ALS Therapy Receives FDA Orphan Drug Designation

The US Food and Drug Administration (FDA) recently granted Orphan Drug Designation to RAG-17, a novel short interfering RNA (siRNA) modality for the treatment of patients with amyotrophic lateral sclerosis (ALS), biopharmaceutical company Ractigen Therapeutics announced.

About 20% of all genetically defined cases of ALS are associated with mutation to the SOD1 gene, the company explained. A therapeutic siRNA, RAG-17 is designed to target and knockdown SOD1 expression in patients with pathogenic mutations that cause ALS.

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The treatment uses Ractigen’s proprietary Smart Chemistry-Aided Delivery platform, in which the siRNA is conjugated to an accessory oligonucleotide, enabling durable and potent activity in central nervous system tissues. In some preclinical studies, RAG-17 had a significantly higher potency on ALS disease models, such as the hSOD1G93A mouse model, than benchmark compounds.

“We are eager to bring RAG-17 to ALS patients as soon as possible, as we believe this therapy can have significantly higher efficacy in patients with the SOD1 mutation compared to the other modalities,” said Ractigen founder, president, and CEO Long-Cheng Li, MD.

The FDA grants orphan drug designation to a drug or biologic intended to treat a rare disease or condition. The designation provides companies with development incentives, including a 7-year marketing exclusivity from the date of market approval and a waiver of the New Drug Application fee.

Reference

Ractigen Therapeutics receives FDA Orphan Drug Designation for the novel oligonucleotide conjugate RAG-17 for the treatment of amyotrophic lateral sclerosis (ALS). News release. Ractigen Therapeutics; March 3, 2023. Accessed March 17, 2023.